By Eleanore Catolico, 10/23/24
Renee Stamper, shown here with a portrait of her mother, has joined 15 clinical trials in eight years. (Photo by Alejandro Ugalde Sandoval)On Christmas Eve 2014, a speeding car clipped the bumper of a Detroit city bus in the early morning darkness. Renee Stamper, the bus driver, spun on the road.
After the accident, Stamper started physical therapy. That’s when she noticed tremors in her left leg. After a few medical exams she was diagnosed with Parkinson’s disease, a chronic nervous system disorder often characterized by tremors.
Her doctor prescribed levodopa, a drug that replaces dopamine that’s lost in the brain due to Parkinson’s and treats the tremors and stiffness in Stamper’s body.
Since then, the 65-year-old has researched her disease online, started boxing to increase her stamina and dedicated her time to help future generations fight the neurological disease that affects nearly 1 million Americans. According to researchers, the national death rate increased from 5.4 per 100,000 people to 8.8 per 100,000 over the last two decades.
Stamper has Medicaid and employer health insurance, so getting Parkinson’s drugs wasn’t an issue. Motivated to learn more about the disease, Stamper said she’s joined 15 clinical trials in eight years.
She later realized her medications couldn’t treat the neuropathy in her feet, so Stamper sought experimental treatments. Each trial tested a single intervention. Stamper remembers taking pills or getting hooked to an IV bag. Across these trials, Stamper said some drugs reduced her symptoms and others didn’t because she was given a placebo. After finishing a trial, she had to wait 90 days to participate in another.
Stamper also sees her choice as an act of medical altruism.
“You can help somebody besides yourself,” Stamper said.
As scientists move to evaluate new medicines that can possibly improve lives, some patients see a clinical trial, which can take months or years to complete and cost millions of dollars, as hope against the perils of disease.
That’s what John Swanson, a pickle farmer; Cindy Finestone, a former respiratory therapist; and Kimi Riegel, a Grosse Pointe resident, discovered, like Stamper, after they joined clinical trials for Parkinson’s treatments.
After her diagnosis, Riegel didn’t attend support groups or anything that gave a glimpse into her new reality.
“I didn’t want to see what it was going to be like for me,” she said.
The math and science of clinical trials
Clinical trials test medical, surgical or behavioral interventions in people. In early studies, researchers are studying the safety and effectiveness of the administered drugs, treatment or devices. Experimental interventions come with risks, ranging from minor discomfort to life-threatening side effects.
While trial volunteers are essential because their reactions to drugs or treatments provide the data researchers need, they are a rare breed. According to a national survey, only 9% of U.S. adults said they’ve been invited to participate in a clinical trial. Within that percentage, 47% joined.
“The actual reason to participate in a trial is because you want to help science move forward,” said Peter H. Schwartz, a professor at Indiana University School of Medicine. “You want to help those who are going to come later have good treatments.”
As disability, illness and premature death caused by neurological conditions rose 18% from 1990 to 2021 globally, there’s more urgency to develop new drugs or therapies, clinical trial experts said.
Without volunteers, clinical research, which experts say is necessary to create equitable health systems and uphold standards of patient care, would be impossible.
Stamper understands her role as volunteer tester, and while she knows there are risks, she says she felt comfortable in the hands of nurses and doctors.
“What they did was reassure me that these trials were safe,” she said.
‘I had no idea what Parkinson’s was’
While sitting at home watching TV, John Swanson felt his first tremor in his toe. As symptoms progressed, his colleagues noticed tremors in his left hand, while working together on Swanson’s pickle farm in Ravenna, a village of 1,300 just outside of Muskegon, Michigan.
In 2012, he and his wife visited a doctor who diagnosed him with Parkinson’s. “We were shocked and just thrown back because I had no idea what Parkinson’s was,” said Swanson, now 65.
Swanson joined a clinical trial at Michigan State University six months after his diagnosis, on a recommendation from his doctor.
This trial tested whether a diabetes drug could reduce Parkinson’s symptoms. He joined a second trial in 2018, which used a repurposed leukemia drug.
Drug repurposing happens when an existing medication labeled to treat one illness is used to treat another. The technique can lower costs associated with clinical trials. One successful example is sildenafil, known on the market as Viagra, which was originally prescribed to treat heart conditions then was repurposed for erectile dysfunction.
Swanson’s physical symptoms are mild. He can still drive. But a few years after his diagnosis, Swanson had “vivid dreams,” at night, common among Parkinson’s patients. “When you wake up, you have to convince yourself they’re not real,” he said.
For the first trial, Swanson picked up the medication to take it home. He trusted his doctor, who helped run the trial. Swanson attended a conference call with researchers and other participants and asked questions about the study.
“I absorbed it like a sponge. This doctor, he just loved to talk about Parkinson’s,” Swanson said. “It was worth my time.”
He said joining clinical trials took away his fears of the disease. Swanson said even though during his second trial, his bedtime dreams stopped, he said he wouldn’t want to give potential trial volunteers false hope.
Sometimes drugs used in clinical trials don’t adequately treat the condition they’re meant to target or the side effects are too strong.
‘It was a horrible day’
Riegel joined a double-blind clinical trial in 2022 at Quest Research Institute in Farmington Hills, Michigan. She hoped a pharmaceutical drug could ease symptoms like walking problems. Neither the patient nor the researcher knew what treatment she ingested until the trial was finished. A placebo or a drug could be pumping through her veins.
Four years earlier, a doctor told Riegel she had Parkinson’s disease.
“It was a horrible day,” said Riegel, now 65.
For 27 days, Riegel swallowed the same unknown pill and slept overnight at the clinic.
Nurses examined her blood samples and her body’s reaction to the treatment dose, which was administered with timed precision. Riegel wasn’t scared of the medications.
“It has never crossed my mind that I would be doing something dangerous,” she said.
As the dosages increased, she got sick. Riegel remembers throwing up and passing out, with nurses and staff members surrounding her to make sure she was OK. That was the only time she had adverse effects in a trial. Overall, her experience was positive.
“I felt supported,” said Riegel, who said her Parkinson’s symptoms lessened as the trial continued.
Testing — and more testing
Clinical trials can be found by consulting a physician, researching opportunities listed in online databases like clinicaltrials.gov or joining a national registry matching potential volunteers to studies.
Trials must comply with U.S. Food and Drug Administration regulations. Institutional review boards, administrative bodies that protect the rights and welfare of human research subjects, monitor safety and ethical standards.
For neurological disease trials, volunteers undergo cognitive assessments, and other testing. They must meet eligibility criteria listed in the protocol, a document that describes the trial’s objectives, rationale, design and other key elements. Criteria can include age, gender or specific health conditions.
“It has never crossed my mind that I would be doing something dangerous.” Kimi Riegel
In recent years, the drug development pipeline for Parkinson’s has included a variety of approaches examining biological aspects of the movement disorder, according to a report published in the Journal of Parkinson’s Disease in July.
Clinical trials have multiple phases. A Phase 1 trial determines the safety, dosage range and the body’s reaction to a drug for a small group of volunteers. Phase 2 further tests the efficacy of a new drug using several hundred volunteers. Phase 3 studies have multiple sites testing a drug on several hundreds to thousands of volunteers, generally the final review before the FDA decides whether to approve a medication for public use. Most drugs don’t make it from Phase 1 to approval.
Drug maker AbbVie recently reported that its experimental drug tavapadon helped improve motor skills and quality of life for early-stage Parkinson’s patients in a Phase 3 trial. Other emerging approaches include a personalized, electric stimulation therapy and the use of diabetes and obesity drugs.
“Right now, wait times to see neurologists and psychiatrists, but especially neurologists, can be really long,” said Leigh Zisko, chief impact officer at the nonprofit Global Alzheimer’s Platform Foundation, which supports clinical trials for Alzheimer and Parkinson’s diseases.
If a patient is in denial, medical testing can validate a diagnosis.
“It’s almost like a second opinion,” Zisko said. “You go through screening for a clinical trial, you almost by default, kind of get another diagnostic workup by that physician. You’re doing everything all over again.”
A lengthy history of exclusion
Most clinical trials for Parkinson’s lack a diverse pool of volunteers, which limits understanding of the disease and undermines public health. New drugs or therapies may prove ineffective for certain racial and ethnic groups.
The exclusion has a long history. The U.S. didn’t establish guidelines to recruit women and people of color until a mandate in 1993. Older adults, medically underserved Black and Latino people, rural residents and people with low incomes remain underrepresented in studies. Some people of color have long lacked trust in the medical system, stemming from the Tuskegee syphilis study.
The country’s racial makeup is 13.7% Black, 6.4% Asian and 19.5% Hispanic or Latino, according to 2023 Census estimates. But in a review of 32,000 people who joined drug trials in 2020, 8% were Black, 6% were Asian and 11% were Latino.
The majority of Parkinson’s studies examine people of European ancestry and it’s unclear how many Black Americans are affected by Parkinson’s and have higher risks for the disease because of their exclusion in genetic research.
What is known is that a Black person is more likely to receive a Parkinson’s diagnosis four years later than a white person.
“Sometimes for the Black community and other underrepresented communities, it’s too late,” said Tamiko MaGee-Rodgers, director of recruitment and strategic initiatives at the Global Alzheimer’s Platform Foundation. “We’ve missed that window.”
In June, the FDA issued draft guidance which requires sponsors to submit diversity action plans for large clinical trials. The guidance includes requirements for sponsors to detail enrollment goals disaggregated by race, ethnicity, gender and age, a rationale for these goals, and recruitment strategies.
Diversifying the pool
As it did in many industries, COVID-19 exposed faults within the clinical trials process and pushed Quest Research to ramp up its diversity efforts.
To bolster engagement with Latino communities, Kara Bardram, the site director, said Quest hopes to hire more Spanish-speaking staff.
Researchers note long-standing barriers can prevent participation, including a lack of transportation, sparse doctor referrals or the absence of a clinical trial site in a specific place. These challenges can have a negative effect on trial recruitment and retention.
To ease volunteer burden, MaGee-Rodgers said she’s seen more clinical trial sponsors decentralize trials, meaning volunteers are not required to regularly visit a research site. Clinical trial sponsors can be companies, organizations or individuals who initiate and oversee a study.
“A nurse may come visit your home and collect that blood versus you going into a site,” she said. “Some other sponsors have mobile units to provide access, go in areas where there are no clinical trial sites or opportunities to participate in clinical trials.”
Finestone, who was diagnosed with Parkinson’s a decade ago, got a package in the mail this year containing a medical device that draws blood from the arm. Clinical researchers wanted to evaluate whether Parkinson’s patients could collect their own blood. At home in Rochester, New York, she used a small box, which resembled an insulin pump, to pierce her skin. “It was pretty self-explanatory,” she said.
Sponsors can provide stipends to volunteers for their time. Compensation can range from $25 to $100 per visit, MaGee-Rodgers said.
Quest Research Institute changed its practices to make participation less inconvenient. Staff members used to hand out bus tickets but pivoted to a more efficient option.
“They were exhausted by the time they got here,” Bardram said. Volunteers now get free Lyft and Uber rides. Stamper drove to the site for trial appointments, and Quest paid for gas.
Trials with fewer tribulations
Riegel, Stamper and Finestone have become patient ambassadors for their communities, well-versed in the possible hazards, disappointments, medical breakthroughs, and chances of hope for people with Parkinson’s.
Stamper discovered 11 classmates from her grade school had Parkinson’s. Then she noticed her twin brother Andre Robbins began to shake and told him to get a neurological exam. Eventually, he got a Parkinson’s diagnosis and began participating in clinical studies.
Parkinson’s hasn’t slowed down Stamper. She’s taken her knowledge of the disease back to her Detroit neighborhood and encourages people who’ve had tremors to get tested for Parkinson’s. She still roller skates, swims and drives a school bus.
Riegel used her stipends from a trial to pay her son’s college loans. She’s given talks about clinical studies and become comfortable with Parkinson’s support groups.
Finestone said she takes several medications, including Azilect and botox injections to alleviate the neck pain caused by cervical dystonia, another symptom of Parkinson’s.
Since her diagnosis, she’s helped some Parkinson’s patients overcome their reticence and fear of trials.
“They don’t want to necessarily admit they have it. I tell them that the more involved you are, then you’ll feel like you have more control over your own illness,” Finestone said.
“If none of us do any of these clinical trials, no one’s going to get better.”
This story was published with the assistance of the Journalism and Women Symposium (JAWS) Health Journalism Fellowship, supported by The Commonwealth Fund.
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